Salih Goran Nadir, Shaker Saher Burhan, Madsen Helle Dall, Bendstrup Elisabeth
Department of Respiratory Medicine, Gentofte University Hospital, Hellerup, Denmark;
Department of Respiratory Medicine, Gentofte University Hospital, Hellerup, Denmark.
Eur Clin Respir J. 2016 Sep 9;3:32608. doi: 10.3402/ecrj.v3.32608. eCollection 2016.
Pirfenidone was approved by the European Medicines Agency and introduced in most European countries in 2011 for treatment of idiopathic pulmonary fibrosis (IPF).
To describe the national Danish experiences of pirfenidone treatment for IPF during 30 months with respect to target population, safety, adherence to the treatment and effect analysis in a well-characterised IPF population in a real-life setting.
Retrospective data collection from medical records of all patients in Denmark with IPF from 2011 to 2014. Data included baseline demographics, high-resolution computed tomography (HRCT), histopathology, forced vital capacity (FVC) and 6-min walk test (6MWT). Longitudinal data on FVC, walk test, adherence to the treatment and vital status were also collected.
Pirfenidone treatment was initiated in 113 patients. Mean age was 69.6±8.1 years (±SD), and 71% were male. Definite IPF diagnosis required thoracoscopic lung biopsy in 45 patients (39.8%). The remaining 68 cases had a definite (64 patients) or possible usual interstitial pneumonia (four patients) pattern on HRCT. Patients were followed for 0.1-33.8 months (median 9.4 months). Fifty-one patients (45.2%) needed dose adjustment, 18 (16%) patients discontinued therapy and 13 patients (11.5%) died. The annual mean decline in FVC was 164 ml (SE 33.2). The decline in 6MWT was 18.2 m (SE 11.2). Nausea (44.2%), fatigue (38.9%) and skin reactions (32.7%) were frequent adverse events.
Patients with IPF treated with pirfenidone experienced tolerable adverse events. Patients were maintained on treatment due to a careful follow-up and dose adjustment programme. The annual decline in physiological parameters and mortality rate was comparable to previous randomised controlled trials.
吡非尼酮于2011年获得欧洲药品管理局批准，并在大多数欧洲国家用于治疗特发性肺纤维化（IPF）。
描述丹麦在30个月内对IPF患者使用吡非尼酮治疗的全国性经验，涉及目标人群、安全性、治疗依从性以及在真实环境中对特征明确的IPF人群的疗效分析。
回顾性收集丹麦2011年至2014年所有IPF患者的病历数据。数据包括基线人口统计学资料、高分辨率计算机断层扫描（HRCT）、组织病理学、用力肺活量（FVC）和6分钟步行试验（6MWT）。还收集了FVC、步行试验、治疗依从性和生命状态的纵向数据。
113例患者开始使用吡非尼酮治疗。平均年龄为69.6±8.1岁（±标准差），71%为男性。45例患者（39.8%）确诊IPF需要进行胸腔镜肺活检。其余68例患者HRCT表现为明确的（64例）或可能的普通型间质性肺炎（4例）模式。患者随访0.1 - 33.8个月（中位数9.4个月）。51例患者（45.2%）需要调整剂量，18例患者（16%）停止治疗，13例患者（11.5%）死亡。FVC的年平均下降量为164 ml（标准误33.2）。6MWT下降量为18.2 m（标准误11.2）。恶心（44.2%）、疲劳（38.9%）和皮肤反应（32.7%）是常见的不良事件。
接受吡非尼酮治疗的IPF患者经历了可耐受的不良事件。通过仔细的随访和剂量调整方案，患者得以维持治疗。生理参数的年下降率和死亡率与先前的随机对照试验相当。
